Method of treating mucopolysaccharidosis type IVA

Abstract

Disclosed are methods and compositions for determining immunodominant peptides of target enzymes used in enzyme replacement therapy for lysosomal storage disorders. More specifically disclosed are immunodominant peptides for N-acetylgalactosamine-6-sulfatase (GALNS). Also disclosed are methods of inducing oral tolerance towards a target enzyme through oral administration of immunodominant peptides prior to commencing enzyme replacement therapy. More specifically disclosed is a method of inducing oral tolerance for GALNS, by orally administering specific immunodominant peptides for GALNS; in subjects suffering from mucopolysaccharidosis type IVA prior to commencing enzyme replacement therapy using GALNS.

What is claimed is: 
     
       1. A method for treating mucopolysaccharidosis type IVA in a subject suffering from mucopolysaccharidosis type IVA, the method comprising: administering to the subject an effective amount of a polypeptide selected from the group consisting of SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, SEQ ID NO:7, SEQ ID NO:8, SEQ ID NO:9, SEQ ID NO:10, SEQ ID NO:11, SEQ ID NO:12 and combinations thereof. 
     
     
       2. The method of  claim 1 , wherein the polypeptide is SEQ ID NO:12. 
     
     
       3. The method of  claim 1 , wherein the effective amount ranges from about 50 μg per administration to about 500 μg per administration. 
     
     
       4. The method of  claim 1 , wherein the polypeptide is administered for 10 weeks. 
     
     
       5. The method of  claim 4 , wherein the polypeptide is administered about every other day for 10 weeks. 
     
     
       6. The method of  claim 1 , wherein the administering is oral administration. 
     
     
       7. The method of  claim 1 , further comprising administering to the subject N-acetyl galactosamine-6-sulfate sulfatase. 
     
     
       8. The method of  claim 7 , wherein the N-acetyl galactosamine-6-sulfate sulfatase is N-acetyl galactosamine-6-sulfate sulfatase enzyme replacement therapy. 
     
     
       9. The method of  claim 7 , wherein the administering is infusion. 
     
     
       10. The method of  claim 7 , wherein the N-acetyl galactosamine-6-sulfate sulfatase ranges from about 50 μg per administration to about 500 μg per administration. 
     
     
       11. The method of  claim 1 , wherein the subject is or has received N-acetyl galactosamine-6-sulfate sulfatase. 
     
     
       12. The method of  claim 11 , wherein the N-acetyl galactosamine-6-sulfate sulfatase is N-acetyl galactosamine-6-sulfate sulfatase enzyme replacement therapy. 
     
     
       13. The method of  claim 11 , wherein the administering is infusion.